Posted on: 1 February 2025, source: evrimagaci.org
On February 1, 2025, the National Institute for Health and Care Excellence (NICE) announced the approval of exagamglogene autotemcel (exa-cel), marking what many are calling a significant breakthrough for the treatment of sickle cell disease. This groundbreaking gene therapy utilizes the CRISPR gene-editing technology, which has transformed medical approaches to genetic disorders, enabling some patients suffering from this debilitating condition to access potentially life-changing treatment.